4 Biotech Companies Working On RNA Therapies

The COVID-19 messenger RNA (mRNA) experimental vaccine from Moderna MRNA 0.53% is one of the most promising vaccine candidates for the disease.

Its success could serve as a proof-of-concept for several other mRNA projects at other publicly-traded companies, such as Pfizer PFE 2.07%, BioNTech BNTX 2.04%, CureVac CVAC 3.49% and Translate Bio TBIO 1.95%.

What Is Messenger RNA (mRNA)?

Our genetic information is stored in DNA molecules in the nuclei of our cells. DNA contains encoded instructions for making various proteins used in the creation or maintenance of cells.

But the ribosomes — the cell organelles which make proteins — can’t actually read DNA. They can only read mRNA, an intermediary form of nucleic acid, which transcribes genetic information from DNA and carries it throughout the cell.

In recent years, scientists have started to experiment with using mRNA as a vector to transmit genetic information to cells, which can teach them how to fight a virus.

In other words, they’ve been looking to use mRNA as a vaccination method.

According to the University of Cambridge’s PHG Foundation, mRNA vaccines could be faster, cheaper and safer to produce than conventional vaccines - which typically contain weakened or modified forms of a pathogen which the patient’s immune system uses as “target practice.”

Moderna’s COVID-19 mRNA Vaccine Candidate

There are currently no mRNA vaccines approved for human use in the U.S. Moderna is looking to change that with MRNA-1273, its mRNA-powered COVID-19 vaccine candidate.

MRNA-1273 is widely viewed as the frontrunner in the race for a COVID-19 vaccine. Moderna reported last week that it has completed enrollment for its 30,000-participant late-stage trial of the vaccine candidate. And unlike Johnson & Johnson JNJ 1.1% or AstraZeneca AZN 0.92%, it has not yet had to halt trials over safety concerns.

If Moderna successfully brings a mRNA vaccine to market — especially if it’s among the first COVID-19 vaccines available to the public — it could bring a surge of interest to mRNA technology in general.

And it’s not the only biotech company working on an mRNA-based project. There are at least three other publicly-traded firms offering investors exposure to this emerging technology.

Pfizer PFE 2.07% And BioNTech’s BNTX 2.04% COVID-19 Vaccine Candidate

Moderna isn’t even the only company working on a COVID-19 vaccine candidate which uses mRNA technology.

Pfizer and BioNTech are also developing BNT162, a mRNA-based COVID-19 vaccine candidate which is currently in global Phase 2/3 trials.

BNT162b2 isn’t quite as far along in the drug development process as MRNA-1723. But it received Fast Track designation from the U.S. Food and Drug Administration (FDA) in July, making it a strong contender to be the second or third mRNA-based vaccine on the market.

CureVac CVAC 3.49% Is Developing Vaccines For Rabies And COVID-19

CureVac is also working on a mRNA-based COVID-19 vaccine, as well as a mRNA-based vaccine for rabies.

The biotecg company has been working on an unnamed mRNA-based COVID-19 vaccine platform along with the nonprofit Coalition for Epidemic Preparedness Innovations (CEPI) since the virus’s genome was published in early January. It is currently in Phase 2 clinical trials.

It developed that platform based on positive early data from its mRNA-based rabies vaccine candidate, CV7202, which is currently in Phase 1 trials.

Translate Bio TBIO 1.95% Is Working On A mRNA-Based Cystic Fibrosis Drug

A new cystic fibrosis drug candidate from Translate Bio provides an example of a non-vaccine drug built on mRNA technology.

Translate Bio recently presented data from its MRT5005 drug candidate at the 34th Annual North American Cystic Fibrosis Conference (NACFC). It’s a mRNA-based gene therapy which seeks to help patients’ malformed lung cells produce essential proteins correctly.

MRT5005, which is currently in Phase 1/2 clinical trials, has also won Orphan Drug, Fast Track and Rare Pediatric Disease designations from the FDA.

https://www.benzinga.com/general/biotech/20/11/18128283/4-biotech-companies-working-on-rna-therapies